Prospective pilot study of a single daily dosage of trientine for the treatment of Wilson disease.

ABSTRACT:

Background

Wilson disease requires lifelong therapy, currently given daily in multiple divided dosages.

Aim

To prospectively evaluate once-daily trientine as therapy for Wilson disease.

Methods

Study group

eight patients (seven males) aged 22-71 years with stable Wilson disease treated from 4 to 50 years. Patients were monitored for 3 months then for 12 months on a single daily dose of trientine (15 mg/kg).

Results

All patients remained clinically well. ALT and AST fluctuated in some, but none required treatment stoppages or side effects. Liver synthetic function was unchanged. Mean 24-h urine copper and zinc excretions at end of treatment were 313.4 ± 191.7 and 2,214 ± 1,346 μg, respectively.

Conclusions

Once-daily trientine should be explored further for possible maintenance therapy for WD. Single daily dose may improve adherence to therapy. Larger trials and longer-term follow-up will establish the safety and treatment efficacy of this once-daily treatment regimen for WD (registration: NCT01472874).

SUBMITTER: Ala A 

PROVIDER: S-EPMC4427615 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature